A Two-Part Study to Investigate the Dose-Ranging Safety and Pharmacokinetics, Followed by the Efficacy and Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution as an Adjunctive Therapy in Children ≥2 Years Old and Young Adults With Dravet Syndrome

Description

Summary

The primary purpose of this study is to evaluate the safety, tolerability and efficacy of a single dose of ZX008 (Fenfluramine Hydrochloride) when added to standard of care and when added to Adjunctive Antiepleptic therapy to Stiripentol treatment in children and young adults with Dravet Syndrome

Official Title

A Multicenter, 2-Cohort Trial to First Assess the Pharmacokinetic and Safety Profile of a Single Dose of ZX008 (Fenfluramine Hydrochloride) Oral Solution When Added to Standard of Care , Followed by a Randomized, Double-blind, Placebo-controlled Parallel Group Evaluation of the Efficacy, Safety, and Tolerability of ZX008 as Adjunctive Antiepileptic Therapy to Stiripentol Treatment in Children and Young Adults With Dravet Syndrome

Details

This is a multicenter, two-cohort trial to assess the pharmacokinetic and safety profile of a single dose of ZX008 (fenfluramine hydrochloride) oral solution when added to Dravet syndrome treatment regimen containing VPA and CLB, with or without STP (Cohort 1), followed by a randomized, double-blind, placebo-controlled parallel group evaluation of the efficacy, safety, and tolerability of ZX008 as adjunctive therapy for seizures in children and young adults with Dravet syndrome (Cohort 2). Cohort 2 will not be dosed until the PK and safety data from Cohort 1 have been collected and evaluated. The PK and safety data from Cohort 1 will determine the dose of ZX008 to be used in Cohort 2.

Keywords

Eligibility

Subject must be male or non-pregnant, non-lactating female, age 2 to 18 years(inclusive)

Subject must have documented medical history to support a clinical diagnosis of Dravet syndrome, where convulsive seizures are not completely controlled by current antiepileptic drugs.

Subject must be receiving a therapeutically relevant and stable dose of CLB, VP, and STP for at least 4 weeks prior to screening and are expected to remain stable throughout the study (Cohort 2 only).

Subject must be receiving a stable dose of CLB and VPA, administered twice daily, to be eligible for Dose Regimen 1 and 2 or subject must be receiving a stable dose of CLB, VPA, and STP, administered twice daily, to be eligible for Dose Regimen 3 (Cohort 1 only).

Key Exclusion Criteria:

Subject has a known hypersensitivity to fenfluramine or any of the excipients in the study medication.

Subject has pulmonary arterial hypertension.

Subject has a current or past history of cardiovascular or cerebrovascular disease,such as cardiac valvulopathy, myocardial infarction or stroke.

Subject has a current or recent history of anorexia nervosa, bulimia, or depression within the prior year that required medical treatment or psychological treatment for a duration greater than 1 month.

Subject is currently taking carbamazepine, oxcarbamazepine, eslicarbazepine,phenobarbital, or phenytoin, or has taken any of these within the past 30 days, as maintenance therapy.

Subject has a positive result on urine THC Panel or whole blood CBD at the Screening Visit.

Subject has a clinically significant condition, or has had clinically relevant symptoms or a clinically significant illness in the 4 weeks prior to the Screening Visit, other than epilepsy, that would negatively impact study participation,collection of study data, or pose a risk to the subject.

Locations

University of California San Franciscoaccepting new patientsSan Francisco, California, 94143, United States